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A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH

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ClinicalTrials.gov Identifier: NCT04544410
Recruitment Status : Recruiting
First Posted : September 10, 2020
Last Update Posted : March 11, 2022
Sponsor:
Information provided by (Responsible Party):
Spruce Biosciences

Brief Summary:
An investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic CAH subjects up to 76 weeks of treatment.

Condition or disease Intervention/treatment Phase
Congenital Adrenal Hyperplasia Drug: Tildacerfont/Placebo Phase 2

Detailed Description:
This is a study that will evaluate the ability of Tildacerfont to reduce the glucocorticoid steroid dose used by adult CAH subjects. The first 24-weeks will be a double-blind, placebo controlled, comparison of Tildacerfont vs Placebo. The following 52-weeks will allow all subjects to move to open label Tildacerfont to continue to reduce steroid dose where appropriate, and observe long term safety.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 90 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Subjects will be randomized in a 1:1 manner to either Tildacerfont or Placebo.
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: Double-Blind
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects With Classic Congenital Adrenal Hyperplasia
Actual Study Start Date : September 29, 2020
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : November 2022


Arm Intervention/treatment
Experimental: Tildacerfont Group
Tildacerfont administered daily via oral tablet for 76 weeks at dose level 1.
Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Name: SPR001

Placebo Comparator: Placebo
Placebo administered daily via oral tablet for 24 weeks.
Drug: Tildacerfont/Placebo
Tablet, administered daily
Other Name: SPR001




Primary Outcome Measures :
  1. Change in GC dose at Week 24 [ Time Frame: 24 Weeks ]
    Absolute change from baseline in GC dose in HCe at Week 24


Secondary Outcome Measures :
  1. Change in GC use adjusted for body surface area in subjects with CAH [ Time Frame: 24 weeks ]
    Absolute change from baseline in GC dose in HCe in mg/m2

  2. Change in the median cumulative HCe dose in subjects with CAH [ Time Frame: 76 Weeks ]
    Median total cumulative GC dose in HCe

  3. Change in GC use while maintaining control of A4 in subjects with CAH [ Time Frame: 24 Weeks ]

    Composite endpoint:

    Absolute change from baseline in GC dose in HCe in subjects who maintain A4 ≤ ULN


  4. Change in supraphysiologic GC use to physiologic GC use while maintaining control of A4 in subjects with CAH [ Time Frame: 24 Weeks ]

    Composite endpoint:

    Proportion of subjects with GC dose ≤20 mg/day in HCe in subjects who maintain A4 ≤ ULN


  5. Change in GC toxicity in subjects with CAH [ Time Frame: 24 weeks ]

    Glucocorticoid Toxicity Index Aggregate Improvement Score (GTI-AIS) (Miloslavsky 2017)

    Score range: -346 to 439 A positive score means worsening and a negative score means improvement


  6. Change in metabolic parameters (fat mass) in subjects with CAH [ Time Frame: 24 weeks ]
    Change from baseline in fat mass as measured by dual-energy X-ray absorptiometry (DXA)

  7. Change in metabolic parameters (body weight) in subjects with CAH [ Time Frame: 24 weeks ]
    Change from baseline in body weight

  8. Change in metabolic parameters (homeostatic model assessment of insulin resistance) in subjects with CAH [ Time Frame: 24 weeks ]
    Change from baseline in homeostatic model assessment of insulin resistance (HOMA-IR)


Other Outcome Measures:
  1. Change in acne in patients with CAH with acne at baseline [ Time Frame: 24 weeks ]
    Change from baseline in Investigator's Global Assessment (IGA) score, a 5 point scale with 0 being a better outcome

  2. Change in acne in patients with CAH with acne at baseline [ Time Frame: 24 weeks ]
    Change from baseline in Subject Numerical Rating Scale (NRS) for acne, an 11-point scale ranging from 0 to 10 with 0 being a better outcome

  3. Change in hirsutism in female CAH subjects with hirsutism at baseline [ Time Frame: 24 weeks ]
    Change from baseline in Modified Ferriman-Gallwey (mFG) score a 5 point scale with 0 being a better outcome Numerical Rating Scale (NRS) for hirsutism

  4. Change in hirsutism in female CAH subjects with hirsutism at baseline [ Time Frame: 24 weeks ]
    Change from baseline in Subject Numerical Rating Scale (NRS) for acne, an 11-point scale ranging from 0 to 10 with 0 being a better outcome



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female subjects over 18 years old, inclusive
  • Has a documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or elevated 17-OHP
  • Has been on a stable, supraphysiologic dose of GC replacement for ≥1 month before screening without any evidence of non-adherence to the GC regimen during this period.
  • For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening

Exclusion Criteria:

  • Has a known or suspected diagnosis of any other known form of classic CAH (not due to 21-hydroxylase deficiency)
  • Has a history that includes bilateral adrenalectomy or hypopituitarism
  • Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist
  • Shows clinical signs or symptoms of adrenal insufficiency

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04544410


Contacts
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Contact: Clinical Trials 415-655-4169 CAH_ClinicalTrials@sprucebiosciences.com

Locations
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Sponsors and Collaborators
Spruce Biosciences
Investigators
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Principal Investigator: Ron Newfield, M.D Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.
Additional Information:
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Responsible Party: Spruce Biosciences
ClinicalTrials.gov Identifier: NCT04544410    
Other Study ID Numbers: SPR001-204
CAHmelia 204 ( Other Identifier: Spruce Biosciences )
First Posted: September 10, 2020    Key Record Dates
Last Update Posted: March 11, 2022
Last Verified: March 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Spruce Biosciences:
CAH
Adrenal Disorder
Congenital Adrenal Hyperplasia
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Hyperplasia
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Congenital Abnormalities
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Gonadal Disorders